Yep, adeno-associated virus (AAV) vectors seem very promising. Depending on the type of vector used, you can target the eye, joints, liver, lungs, etc. [1]. Rel

Yep, adeno-associated virus (AAV) vectors seem very promising. Depending on the type of vector used, you can target the eye, joints, liver, lungs, etc. [1]. Relatively recently, the FDA approved the first AAV-based therapy for a rare retinal disorder [2]. Also worth checking out are some promising trials for treatment of severe hemophilia using AAV vectors [3]. These might end up being great, because the current gold standard treatment involves giving people regular injections of recombinant clotting factor protein, which is even more expensive than gene therapy. Plus, with gene therapy, you have one treatment that lasts a long time, versus having to get an infusion of the treatment regularly. [1] (https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5138125/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5138125/) [2] (https://www.labroots.com/trending/drug-discovery/7533/adeno-associated-virus-gene-therapy-fda-approved-rare-retinal-disease https://www.labroots.com/trending/drug-discovery/7533/adeno-...) [3] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5728634/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5728634/